Drug intelligence / Profile preview

siRNA targeting RRM2

Development stage
Preclinical
Lead developer
Calando Pharmaceuticals
Modality
Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intravenous
01

Overview

siRNA targeting RRM2 refers to a class of RNA interference (RNAi) therapeutics designed to silence the expression of the M2 subunit of ribonucleotide reductase (RRM2). RRM2 is a rate-limiting enzyme essential for the production of deoxyribonucleoside triphosphates (dNTPs) required for DNA synthesis and repair. Its overexpression is frequently observed in various malignancies, including lung, head and neck, and pancreatic cancers, where it is associated with increased tumor cell proliferation, invasiveness, and resistance to chemotherapy. The most prominent clinical-stage asset in this category was CALAA-01, developed by Calando Pharmaceuticals. CALAA-01 was a targeted nanoparticle-siRNA therapeutic consisting of a cyclodextrin-containing polymer delivery system decorated with transferrin ligands to facilitate uptake by transferrin receptor-overexpressing tumor cells. Although CALAA-01 provided the first clinical proof-of-concept for systemic siRNA delivery and gene silencing in humans, its clinical development was ultimately discontinued after Phase 1 trials.

Other names
RRM2 siRNARRM-2 siRNARRM 2 siRNAsiRRM2siRRM-2siRRM 2siRNA targeting ribonucleotide reductase subunit M2
02

Targets

RRM2

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